Clene Announces Publication Describing CNM-Au8 Brain Target Engagement in Neurodegenerative Diseases

Clene Inc. and its wholly owned subsidiary Clene Nanomedicine Inc. recently announced the publication of a peer-reviewed article describing brain target engagement by CNM-Au8, the company’s lead drug candidate. CNM-Au8 is an orally delivered suspension of clean-surfaced, catalytically active gold nanocrystals shown to have neuroprotective and neuroreparative properties in multiple preclinical models of neurodegenerative disease.

The paper, titled Evidence of Brain Target Engagement in Parkinson’s Disease and Multiple Sclerosis by the Investigational Nanomedicine, CNM-Au8, in the REPAIR Phase 2 Clinical Trials, was published in the Journal of Nanobiotechnology, a Springer Nature journal that communicates significant advances in the fields of medicine and biology with an emphasis in their interface with nanoscale sciences. The article is available via Open Access at: https://jnanobiotechnology.biomedcentral.com/articles/10.1186/s12951-023-02236-z.

Co-authored by physician scientists from the University of Texas Southwestern (UTSW) Medical Center and Clene, the publication describes results from two Phase 2a clinical trials, REPAIR-PD and REPAIR-MS. Key brain metabolites, such as NAD+ and NADH, that are involved in neuronal energy production, utilization, and maintenance, were measured for changes from baseline with daily, oral treatment of CNM-Au8 over 12 weeks. The studies’ primary endpoint, the change in brain ratio of the metabolites NAD+ to NADH from baseline, was met with statistical significance (p < 0.05) by a demonstrated 10.4% increase. Statistically significant treatment effects were also observed for secondary and exploratory imaging outcomes, including favorable effects on brain ATP (adenosine triphosphate) levels and phosphorylation potential across both cohorts.

Dr. Benjamin Greenberg, Head of Medical at Clene, said “We believe the 10.4% increase in brain NAD+/NADH ratio to be clinically significant. Other groups have shown significant deficits in brain energy metabolites associated with neurodegenerative disease. For example, brain levels of NAD are deficient in Parkinson’s disease, and deficits in brain ATP levels correlate with the Expanded Disability Status clinical scale for multiple sclerosis. Even in healthy aging, the human brain’s NAD+/NADH decreases at a rate of loss of approximately half a percent per decade. Elevation of brain NAD+/NADH levels to many times the rate of loss observed in healthy aging is a significant and very promising effect.”

Clene Inc., (Nasdaq: CLNN) and its wholly owned subsidiary Clene Nanomedicine Inc., is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis, Parkinson’s disease and multiple sclerosis. CNM-Au8 is an investigational first-in-class therapy that improves central nervous system cells’ survival and function via a mechanism that targets mitochondrial function and the NAD pathway while reducing oxidative stress. CNM-Au8 is a federally registered trademark of Clene Nanomedicine, Inc. The company is based in Salt Lake City, UT, with R&D and manufacturing operations in MD. For more information, visit www.clene.com.